The US FDA has accepted the NDA for nirogacestat in adults with desmoid tumors. The application also granted priority review with an expected PDUFA date of Aug 27, 2023
The NDA was based on the P-III trial (DeFi) results evaluating nirogacestat (150mg) vs PBO in a ratio (1:1) in 142 patients. The 1EPs were PFS or death by any cause while the 2EPs & exploratory EPs incl. safety & tolerability measures, ORR, DoR, changes in tumor volume as assessed by MRI & changes in PROs
Nirogacestat is an oral, selective, small molecule gamma-secretase inhibitor & has received FTD & BTD from the US FDA for progressive, unresectable, recurrent, or refractory desmoid tumors or deep fibromatosis; ODD from the US FDA for desmoid tumors & from EC for soft tissue sarcoma
Ref: Globenewswire | Image: SpringWorks Therapeutics
Related News:- SpringWorks Reports the Completion of NDA Submission to the US FDA for Nirogacestat to Treat Desmoid Tumors
